Ursodeoxycholic acid treatment in non-alcoholic steatohepatitis patients due to familial hypobetalipoproteinemia

Öz

Background/aim: Hypobetalipoproteinemia (HBL) is a congenital lipid metabolism disorder. The prognosis and treatment of fatty liver in this disease are not clearly known. Herein we report a prospective pilot study we conducted in order to determine the efficacy of ursodeoxycholic acid treatment in patients with non-alcoholic steatohepatitis (NASH) due to familial HBL (FHBL). Materials and methods: The study included three patients with NASH due to FHBL. The diagnosis of NASH was based on abnormal serum transaminases for more than six months; a liver biopsy showing the presence of steatosis, ballooning degeneration, and necroinflammation with or without Mallory bodies, fibrosis, or cirrhosis; and appropriate exclusion of other liver diseases. The diagnosis of FHBL was based on < 5th percentile plasma levels of total cholesterol or LDL cholesterol, or total apolipoprotein B (apoB), demonstration of low levels of LDL or apoB in one of the first-degree relatives, and exclusion of secondary causes. Patients received ursodeoxycholic acid (15mg/kg/day) for 12 months. Serum transaminases were followed every three months. Results: The study group included one female (44 years old) and two male (22 and 38 years old) patients. All patients were asymptomatic. Liver biopsy revealed mild inflammation and fibrosis in all patients. None of the patients had ALT normalization at the end of one year of treatment with ursodeoxycholic acid. Conclusion: Ursodeoxycholic acid is not an effective drug in the treatment of NASH due to FHBL.

Anahtar Kelimeler

• Steatohepatitis, hypobetalipoproteinemia, ursodeoxycholic acid

Ailesel hipobetalipoproteinemiye bağlı non-alkolik steatohepatitli hastalarda ursodeoksikolik asit tedavisi

Öz

Giriş ve amaç: Hipobetalipoproteinemi (HBL) kalıtsal bir lipid metabolizma hastalığı olup bu hastalarda görülebilen karaciğer yağlanmasının prognoz ve tedavisi henüz net olarak bilinmemektedir. Bu çalışmada ailevi hipobetalipoproteinemiye bağlı non-alkolik steatohepatitli (NASH) hastalarda ursodeoksikolik asit tedavisinin etkinliğini araştırılması amacı ile prospektif pilot bir çalışma yürütülmüştür. Gereç ve yöntem: Çalı şmaya ailevi hipobetalipoproteinemiye bağlı NASH tanısı konulan 3 hasta alındı. NASH tanısı 6 aydan uzun süredir serum transaminazlarında yükselme, karaciğer biyopsisinde steatoz, balon dejenerasyon ve nekroinflamasyona eşlik edebilen Mallory cisimcikleri, fibrozis veya siroz varlığı ve diğer karaciğer hastalıklarının ekarte edilmesi ile konuldu. Ailevi hipobetalipoproteinemi tanısı hastanın total kolesterol veya LDL veya total apolipoprotein B (apoB) düzeylerinin 5 persentilin altına inmesi, birinci dereceden yakınlarından birinde LDL veya apoB düşüklüğünün gösterilmesi ve sekonder nedenlerin ekarte edilmesi ile konuldu. Hastalara 12 ay süre ile 15mg/kg/gün dozunda ursodeoksikolik asit tedavisi verildi. Üç ayda bir serum transaminaz düzeyleri takip edildi. Bulgular: Çalışmaya alınan 3 hastanın 2'si 23 ve 38 yaşlarında erkek, 1'i 44 yaşında kadındı. Hepsi asemptomatikti. Karaciğer biyopsisinde tüm hastalarda hafif düzeyde inflamasyon ve fibrozis mevcuttu. Hastaları n hiçbirinde bir yıllık ursodeoksikolik asit tedavisi sonunda serum ALT değerlerinde normalleşme görülmedi. Sonuç: Ursodeoksikolik asit ailevi hipobetalipoproteinemiye bağlı NASH'ın tedavisinde etkin bir tedavi değildir.

Anahtar Kelimeler

• Steatohepatit, hipobetalipoproteinemi, ursodeoksikolik asit

Kaynakça

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